Bluebird Withdraws Zynteglo from German Market, Cuts Workforce

Rare Daily Staff

Bluebird Bio said it is withdrawing its gene therapy Zynteglo for transfusion-dependent beta-thalassemia from the German market over pricing and is embarking on a targeted reshaping of its workforce intended to enable the company to advance its late-stage gene therapy programs.

The company said reimbursement negotiations in Germany did not result in a price for Zynteglo that it believes reflects the value of this one-time gene therapy with potential life-long benefit for people living with transfusion-dependent beta-thalassemia (TDT). The price proposed by the German health authorities fails to recognize the severe burden of living with TDT or the innovation and benefit Zynteglo brings to patients who are impacted every day, throughout their lives by this severe genetic disease.

As part of an update, the company also said that a previously reported case of myelodysplastic syndrome in its phase 1/2 study of HGB-206, its LentiGlobin gene therapy for sickle cell disease, has been further assessed following the review of results from additional tests. The treating investigator has concluded this is not a case of MDS and has revised the diagnosis to transfusion-dependent anemia.

Bluebird Bio has reported this update to regulatory agencies and study investigators. The company continues to work with the treating investigator to determine the potential cause of this patient’s anemia.

HGB-206 is an open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for SCD that includes three treatment cohorts. A refined manufacturing process designed to increase vector copy number and further protocol refinements made to improve engraftment potential of gene-modified stem cells were used for the third cohort known as Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.

Last month, the company reported that it is very unlikely the suspected unexpected serious adverse reaction of acute myeloid leukemia reported in the HGB-206 study of LentiGlobin for SCD was related to the BB305 lentiviral vector. This assessment, along with the re-classification of the originally reported MDS case to transfusion-dependent anemia are important steps in Bluebird Bio’s path to seeking removal of the clinical hold on studies HGB-206 and HGB-210 of LentiGlobin for SCD.

Bluebird said it continues to work with regulators to resume its clinical studies in sickle cell disease as well as to remove the clinical hold for HGB-207 and HGB-212 clinical studies of beti-cel for β-thalassemia, with potential lift of all clinical holds in mid-2021.

In response to these and other events and shifts related to the business over the past year, Bluebird Bio also said it plans to reduce its workforce, primarily in Europe. It did not say how big a reduction in staff it is making. The reduction and reallocation of resources is intended to focus on priority European markets and streamline global operations going forward to ensure its ability to deliver gene therapies to patients based on Bluebird Bio’s current business plans.